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DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype

Antisense oligonucleotide (AON)‐mediated exon skipping is an emerging therapeutic for individuals with Duchenne muscular dystrophy (DMD). Skipping of exons adjacent to common exon deletions in DMD using AONs can produce in‐frame transcripts and functional protein. Targeted skipping of DMD exons 8, 4...

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Detalles Bibliográficos
Autores principales:	Wang, Richard T., Barthelemy, Florian, Martin, Ann S., Douine, Emilie D., Eskin, Ascia, Lucas, Ann, Lavigne, Jenifer, Peay, Holly, Khanlou, Negar, Sweeney, Lee, Cantor, Rita M., Miceli, M. Carrie, Nelson, Stanley F.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2018
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6175390/ https://www.ncbi.nlm.nih.gov/pubmed/29907980 http://dx.doi.org/10.1002/humu.23561

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